News

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…

The first two patients have been enrolled in the high-dose group of a U.S. clinical trial testing the safety and efficacy of AMT-130 as a potential gene therapy for Huntington’s disease. “We are very pleased with the progress that we continue to make in this Phase 1/2 clinical trial and…

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…

Galyan Bio plans to initiate a clinical trial of a small molecules candidate that aims to slow the progression of Huntington’s disease next year, working in with Bicoll GmbH. Galyan Bio is developing and optimizing small molecules as oral treatments of Huntington’s and other neurodegenerative disorders, which work by targeting disease-causing mutant…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

Metabolites seen as potential biomarkers of Huntington’s disease — called kynurenine pathway metabolites — showed little evidence of Huntington’s-specific activity in a recent study. While researchers could not entirely rule out kynurenine pathway metabolites as possible treatment targets, their results suggest that the disease is not well represented by the…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

A Phase 1/2 trial testing the safety and efficacy of AMT-130, uniQure’s experimental gene therapy for Huntington’s disease, can continue dosing after a positive recommendation from an independent board. The data safety monitoring…

Prilenia Therapeutics has enrolled more than 120 patients in PROOF-HD, a Phase 3 clinical trial investigating the efficacy and safety of oral pridopidine in early stage Huntington’s disease. That amounts to 25% of the target enrollment of up to 480 patients, the company announced. The trial is…

uniQure has completed patient enrollment for the first group participating in a Phase 1/2 trial assessing the safety and efficacy of AMT-130, its experimental gene therapy for Huntington’s disease…